Sickle cell drug Endari wins FDA approval; first new drug in 20 years

This 2009 colorized microscope image made available by the Sickle Cell Foundation of Georgia via the Centers for Disease Control and Prevention shows a sickle cell, left, and normal red blood cells of a patient with sickle cell anemia.

Janice Haney Carr, AP

The U.S. Food and Drug Administration has approved the first drug in nearly 20 years for sickle cell, an inherited disease in which abnormally shaped red blood cells can't properly carry oxygen throughout the body, which can cause severe pain and organ damage.

About 100,000 people in the U.S. have the disorder and about 275,000 babies are born with it each year worldwide. The disease mainly affects African-Americans, Latinos and other minority groups.

In a study, the new drug, Endari, was tested in patients ages 5 to 58 years old with sickle cell disease who had two or more painful crises within the past 12 months. The participants were randomly given treatment with Endari or a placebo and evaluated over the course of 48 months.

The results showed that Endari cut the number of pain crises and dangerous chest complications, and reduced hospitalizations and the need for transfusions.

"Endari is the first treatment approved for patients with sickle cell disease in almost 20 years," Dr. Richard Pazdur, M.D., acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research and director of the FDA's Oncology Center of Excellence, said in a statement. "Until now, only one other drug was approved for patients living with this serious, debilitating condition."

The FDA says common side effects of Endari include constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain and chest pain.

It is made by a California company, Emmaus Medical Inc., and approved for adults and children 5 and older.